Santhera Pharmaceuticals, a Swiss pharmaceutical company specialized in developing and commercializing breakthrough treatments for orphan mitochondrial and neuromuscular diseases, announced that it has dosed the first patient in its CALLISTO Phase I trial that aims to evaluate the safety and tolerability profile of orally-stable omigapil for Congenital Muscular Dystrophy (CMD). All necessary participants have also been screened and enlisted for the study.
CALLISTO is currently underway at the National Institutes of Health (NIH). Aside from determining the drug’s safety and tolerability profile, it will also assess the possibility of conducting disease-specific clinical assessments that could be used as clinical study endpoints in pediatric and adolescent efficacy trials down the road. At present, Omigapil is a pipeline drug in-licensed by Novartis and repositioned by Santhera for CMD.
CALLISTO will be studying the effects of the drug in 20 ambulatory and non-ambulatory CMD patients aged 5 to 16 years old. They have been randomized to 3 cohorts, each to receive different ascending doses of omigapil (0.02, 0.08 or 0.2 mg/kg/day) over a course of 12 weeks. Investigators will be taking note of the participants’ respiratory function, muscle strength and motor function. Santhera expects the study will run until late 2016.
“We are very excited about the start of this trial with the first investigational new drug for this group of severe neuromuscular diseases where children are affected with ultimately devastating loss of functional muscle and no treatment currently available to slow down or stop progression of the disease”, commented Carsten Bonnemann, MD, a senior investigator in the NINDS Neuromuscular and Neurogenetic Disorders of Childhood Section and Principal Investigator of this study. “To ensure as much information as possible can be collected from patients with both Ullrich or MDC1A subtypes of CMD in this trial, it was important to have all patients identified and randomized prior to the first patient commencing treatment.”
“We are very proud of being able to collaborate with the NIH in developing omigapil for CMDs”, emphasized Thomas Meier, PhD, Chief Executive Officer of Santhera. “We are thankful to the patients and families who are making tremendous efforts to enroll into CALLISTO, which is the first intervention trial with an investigational medicinal product in this group of neuromuscular diseases.”